Refractory Thrombocytopenia is the Earliest Diagnostic Criterion for Sinusoidal Obstruction Syndrome in Children.


Journal

Journal of pediatric hematology/oncology
ISSN: 1536-3678
Titre abrégé: J Pediatr Hematol Oncol
Pays: United States
ID NLM: 9505928

Informations de publication

Date de publication:
26 Aug 2024
Historique:
received: 24 01 2024
accepted: 16 07 2024
medline: 26 8 2024
pubmed: 26 8 2024
entrez: 26 8 2024
Statut: aheadofprint

Résumé

Sinusoidal obstruction syndrome (SOS) is a life-threatening complication of hematopoietic stem cell transplantation (HSCT), whose diagnostic criteria changed over time to achieve a timelier diagnosis. Recently, pediatric-specific criteria presented by the European Society for Blood and Marrow Transplantation (pEBMT) incorporated transfusion-refractory thrombocytopenia (RT) as an early indicator of SOS in children. However, a comparison of all individual diagnostic parameters belonging to pEBMT and former SOS diagnostic criteria has never been performed. This retrospective study conducted at a pediatric tertiary care hospital analyzed all pediatric HSCT cases diagnosed with SOS among 170 children transplanted from 2009 to 2023. Eleven patients developed SOS during this period (incidence: 11/170, 6.5%). pEBMT, Seattle, and Baltimore criteria were retrospectively applied to the 11 cases and compared, showing that RT was the earliest fulfilled parameter (median onset: 6 d post-HSCT). pEBMT and Seattle criteria identified 11/11 SOS cases, with pEBMT leading to an earlier diagnosis. RT typically manifested before diagnosis, with significantly higher platelet transfusion requirements before diagnosis than after. RT is the earliest satisfied criterion in pediatric SOS and typically occurs in the initial stages of the disease before diagnosis. Further research is needed to identify additional early indicators of pediatric SOS.

Identifiants

pubmed: 39185856
doi: 10.1097/MPH.0000000000002938
pii: 00043426-990000000-00485
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Informations de copyright

Copyright © 2024 The Author(s). Published by Wolters Kluwer Health, Inc.

Déclaration de conflit d'intérêts

The authors declare no conflicts of interest.

Références

Mohty M, Malard F, Abecasis M, et al. Prophylactic, preemptive, and curative treatment for sinusoidal obstruction syndrome/veno-occlusive disease in adult patients: a position statement from an international expert group. Bone Marrow Transplant. 2020;55:485–495.
Corbacioglu S, Carreras E, Ansari M, et al. Diagnosis and severity criteria for sinusoidal obstruction syndrome/veno-occlusive disease in pediatric patients: a new classification from the European society for blood and marrow transplantation. Bone Marrow Transplant. 2018;53:138–145.
Bonifazi F, Barbato F, Ravaioli F, et al. Diagnosis and treatment of VOD/SOS after allogeneic hematopoietic stem cell transplantation. Front Immunol. 2020;11(April):1–13.
Corbacioglu S, Greil J, Peters C, et al. Defibrotide in the treatment of children with veno-occlusive disease (VOD): a retrospective multicentre study demonstrates therapeutic efficacy upon early intervention. Bone Marrow Transplant. 2004;33:189–195.
Kernan NA, Grupp S, Smith AR, et al. Final results from a defibrotide treatment-IND study for patients with hepatic veno-occlusive disease/sinusoidal obstruction syndrome. Br J Haematol. 2018;181:816–827.
Richardson PG, Smith AR, Kernan NA, et al. Pooled analysis of Day 100 survival for defibrotide-treated patients with hepatic veno-occlusive disease/sinusoidal obstruction syndrome and ventilator or dialysis dependence following haematopoietic cell transplantation. Br J Haematol. 2020;190:583–587.
Jones RJ, Kamthorn SKL, Beschorner WE, et al. Venoocclusive disease of the liver following bone marrow transplantation. Transplantation. 1987;44:778–783.
McDonald GB, Hinds MS, Fisher LD, et al. Veno-occlusive disease of the liver and multiorgan failure after bone marrow transplantation: a cohort study of 355 patients. Ann Intern Med. 1993;118:255–267.
Corbacioglu S. The coming of age of the pediatric EBMT criteria. Bone Marrow Transplant. 2021;56:767–768.
Embaby MM, Rangarajan HG, Abu-Arja R, et al. Refractory thrombocytopenia is a valid early diagnostic criteria for hepatic veno-occlusive disease in children. Biol Blood Marrow Transplant. 2020;26:546–552.
Nash RA, Gooley T, Davis C, et al. The problem of thrombocytopenia after hematopoietic stem cell transplantation. Stem Cells. 1996;14:(suppl 1)261–273.
Young JA, Pallas CR, Knovich MA. Transplant-associated thrombotic microangiopathy: theoretical considerations and a practical approach to an unrefined diagnosis. Bone Marrow Transplant. 2021;56:1805–1817.
Bento L, Bastida JM, García-Cadenas I, et al. Thrombopoietin receptor agonists for severe thrombocytopenia after allogeneic stem cell transplantation: experience of the Spanish group of hematopoietic stem cell transplant. Biol Blood Marrow Transplant. 2019;25:1825–1831.
Schwartz GJ, Muñoz A, Schneider MF, et al. New equations to estimate GFR in children with CKD. J Am Soc Nephrol. 2009;20:629–637.
Mahadeo KM, Bajwa R, Abdel-Azim H, et al. Diagnosis, grading, and treatment recommendations for children, adolescents, and young adults with sinusoidal obstructive syndrome: an international expert position statement. Lancet Haematol. 2020;7:e61–e72.
Caselli D, Cimaz R, Besio R, et al. Partial rescue of biochemical parameters after hematopoietic stem cell transplantation in a patient with prolidase deficiency due to two novel PEPD mutations. JIMD Rep. 2012;3:71–77.
Consonni F, Dotta L, Todaro F, et al. Signal transducer and activator of transcription gain-of-function primary immunodeficiency/immunodysregulation disorders. Curr Opin Pediatr. 2017;29:1.
Consonni F, Favre C, Gambineri E. IL-2 signaling axis defects: how many faces? Front Pediatr. 2021;9:669298.
Ragoonanan D, Khazal SJ, Wang J, et al. Improved detection of sinusoidal obstructive syndrome using pediatric–AYA diagnostic criteria and severity grading. Bone Marrow Transplant. 2021;56:175–184.
Füssiová M, Švec P, Horáková J, et al. The Importance of new EBMT criteria on the diagnosis of veno-occlusive liver disease in children. J Clin Med. 2023;12:826.
Faraci M, Bertaina A, Luksch R, et al. Sinusoidal obstruction syndrome/veno-occlusive disease after autologous or allogeneic hematopoietic stem cell transplantation in children: a retrospective study of the Italian Hematology-Oncology Association–Hematopoietic Stem Cell Transplantation Group. Biol Blood Marrow Transplant. 2019;25:313–320.
Dhir A, Wadhwa A, Haines H, et al. Retrospective application of sinusoidal obstruction syndrome/veno-occlusive disease diagnostic criteria in a pediatric hematopoietic stem cell transplant cohort. J Pediatr Hematol Oncol. 2022;44:E343–E348.
Cesaro S, Pillon M, Talenti E, et al. A prospective survey on incidence, risk factors and therapy of hepatic veno-occlusive disease in children after hematopoietic stem cell transplantation. Haematologica. 2005;90:1396–1404.
Zama D, Bossù G, Ravaioli F, et al. Longitudinal evaluation of liver stiffness in three pediatric patients with veno-occlusive disease. Pediatr Transplant. 2019;23:e13456.
Debureaux PE, Bourrier P, Rautou PE, et al. Elastography improves accuracy of early hepato-biliary complications diagnosis after allogeneic stem cell transplantation. Haematologica. 2021;106:2374–2383.

Auteurs

Filippo Consonni (F)

Departments of Experimental and Clinical Biomedical Sciences "Mario Serio".
Centre of Excellence, Division of Pediatric Oncology/Hematology, Meyer Children's Hospital IRCCS, Florence, Italy.

Alice Ciulli (A)

Health Sciences.

Daniela Cuzzubbo (D)

Centre of Excellence, Division of Pediatric Oncology/Hematology, Meyer Children's Hospital IRCCS, Florence, Italy.

Stefano Frenos (S)

Centre of Excellence, Division of Pediatric Oncology/Hematology, Meyer Children's Hospital IRCCS, Florence, Italy.

Maria Chiara Sanvito (MC)

Centre of Excellence, Division of Pediatric Oncology/Hematology, Meyer Children's Hospital IRCCS, Florence, Italy.

Annalisa Tondo (A)

Centre of Excellence, Division of Pediatric Oncology/Hematology, Meyer Children's Hospital IRCCS, Florence, Italy.

Veronica Tintori (V)

Centre of Excellence, Division of Pediatric Oncology/Hematology, Meyer Children's Hospital IRCCS, Florence, Italy.

Eleonora Gambineri (E)

Neurosciences, Psychology, Drug Research and Child Health (NEUROFARBA), University of Florence.
Centre of Excellence, Division of Pediatric Oncology/Hematology, Meyer Children's Hospital IRCCS, Florence, Italy.

Classifications MeSH